GD03 Identifying core outcome domains and development of a cutaneous leishmaniasis core outcome measure for use in clinical practice and trials

Abstract

Cutaneous leishmaniasis (CL) is caused by an intracellular protozoan parasite transmitted through a sand fly bite. A review of clinical trials examining different treatment modalities for CL found studies to be deficient in design, execution, analysis and reporting. Core outcome sets (COS), including agreed domains and measures, have been developed for several dermatological disorders. The aims of this study were to reach consensus on the most important core outcome domains to measure when assessing CL, agree on parameters to measure the domains and develop a clinical tool to assess CL. Potential outcome domains for CL were identified through a collaborative international approach involving virtual discussions and e-consultations with stakeholders, including teams caring for CL from Sri Lanka, India, Brazil and the UK. A subsequent judgement process was then undertaken, using the multidisciplinary stakeholder panel of international experts, adhering to nominal group technique to reach final agreement on the core outcome domains. Clinicians presented cases representing the clinical and psychosocial impact of CL to ensure the patient’s perspective was considered. Established core outcome domains included signs and symptoms (objective and subjective), treatment efficacy, treatment impact, clinical sequelae (scarring and pigment) and health-related quality of life (HRQoL). For each core outcome domain, a review of previous approaches to measure the domain was considered. Items to measure the domains were then evaluated by multiple rounds until the panel of experts reached consensus. It was agreed that ‘signs and symptoms’ should capture clinical morphology, diameter and induration of an index lesion with the aid of a palpability score; ‘treatment efficacy and impact’ should capture patient and investigator views through visual analogue scores, percentage change in size of the lesion and re-epithelialization compared with baseline. Sequelae assessment rated pigment, as well as atrophic and hypertrophic/keloid scars. Two open-ended questions were included as a means of capturing some aspects of HRQoL. CutLeishCOM was then generated to capture demographic details, reflect the core outcome domains and assess outcome measurements in each patient. CutLeishCOM has been validated in a small cohort in this study; further validation is required. A specific patient-reported outcome measure should also be considered to ensure adverse effects from treatments, and negative impacts are captured when assessing CL, thus enabling a patient-focused and empathic approach to management.